WASHINGTON, Dec 6 — In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering — by editing her genome. Victoria Gray’s recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one...
First unveiled about six years ago, CRISPR-Cas9 allows scientists to insert, remove and correct a faulty sequence on a strand of DNA in a cell with pinpoint precision. — poba/Istock.com pic via AFP
The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 — pronounced “Crisper” — a new tool informally known as molecular “scissors”. “It’s all developing very quickly,” said French geneticist Emmanuelle Charpentier, one of Crispr’s inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.
“We have hit an inflection point,” said Carrie Wolinetz, NIH’s associate director for science policy.
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